RPGR gene therapy for retinitis pigmentosa

US10314924B2 · kind B2 · utility

Assignees

• Massachusetts Eye and Ear Infirmary · US
• UCL BUSINESS PLC · GB
• The United States of America, as represented by the Secretary, Department of Health and Human Services · US

Inventors

• Michael Sandberg · Mölndal, SE
• Basil Pawlyk · Exeter, US
• Alan Wright · Irving, US
• Xinhua Shu · Glasgow, GB
• Tiansen Li · Quincy, US
• Robin Ali · London, GB

Key dates

• Filing date: Jul 17, 2015
• Grant date: Jun 11, 2019
• Expiry date: Jul 17, 2035

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14143
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Methods for treating a human subject who has X-linked Retinitis Pigmentosa (XLRP) or another clinically-defined ophthalmological condition due to a loss-of-function mutation in the gene encoding the retinitis pigmentosa GTPase regulator (RPGR) protein, the method comprising administering to the subject a nucleic acid comprising an adeno-associated viral vector comprising an abbreviated human RPGR cDNA.